A new Pittsburgh-based venture philanthropy platform unites EB Research Partnership, founded by Jill Vedder and Eddie Vedder of Pearl Jam, with the University of Pittsburgh Health Sciences, UPMC Children’s Hospital of Pittsburgh, UPMC Vision Institute, Carnegie Mellon University, Stanford Medicine, and ElevateBio to create a scalable engine combining artificial intelligence, clinical research, therapeutic development, and commercialization to accelerate treatments and cures for rare diseases.
PITTSBURGH, PA — (August 12, 2026) The Richard King Mellon Foundation has made a landmark investment of up to $25 million to launch Rare Ventures ™, a first-of-its-kind venture philanthropy platform designed to accelerate the development of therapies for rare diseases and establish a new model for medical innovation.
The announcement marks one of the largest philanthropic commitments dedicated to building a new model for rare disease innovation. Rare Ventures is an ambitious effort to do for rare-disease medicine what venture capital did for technology: creating the infrastructure, capital, expertise, and partnerships required to transform promising ideas into accessible treatments for the hundreds of million people suffering from rare diseases without hope of a cure.
Founded by the team behind leading patient foundation EB Research Partnership (EBRP) and built in collaboration with the University of Pittsburgh Health Sciences, UPMC Children’s Hospital of Pittsburgh, UPMC Vision Institute, Carnegie Mellon University, Stanford Medicine, ElevateBio and leading biotechnology partners, Rare Ventures seeks to transform how discoveries become treatments by integrating artificial intelligence, patient data, basic and translational research, clinical development, manufacturing, and commercialization into a single coordinated platform.
More than 400 million people worldwide live with a rare disease, yet approximately 95% of the more than 10,000 known rare diseases have no approved treatment. Rare diseases affect more people than cancer. While scientific breakthroughs in genomics, gene editing, artificial intelligence, and biotechnology have accelerated dramatically over the past decade, the systems required to translate those discoveries into therapies at scale have not kept pace.
Rare Ventures was created to change that.
Rare Ventures builds upon the leading patient foundation EBRP’s model that has demonstrated success in the life- threatening genetic rare skin disease epidermolysis bullosa (EB) in one of medicine’s most challenging fields: rare disease. Over the past 14 years, EBRP has helped fund more than 180 research projects in 22 countries, contributed to the expansion of the EB clinical trial landscape from two active trials to more than 50, and accelerated the development of three FDA-approved therapies in the last three years. Through its pioneering venture philanthropy approach, EBRP has also demonstrated that financial returns generated from successful therapies can be reinvested into future research, creating a sustainable cycle of innovation and impact.
Rare Ventures expands that model beyond a single disease.
The platform brings together three integrated pillars designed to address the most significant barriers in rare disease development: AI-powered patient data infrastructure, venture philanthropy investment, and therapeutic development through basic research, clinical translation, regulatory strategy, manufacturing, and commercialization. Together, these capabilities create a repeatable framework for advancing therapies from discovery to patients faster, more efficiently, and at greater scale.
The initiative launches with an initial portfolio spanning multiple rare diseases and is designed to grow into a platform capable of supporting hundreds of disease communities over time.
“Rare disease patients have waited long enough,” said Michael Hund, MBA, Chief Executive Officer of EB Research Partnership and Co-Founder of Rare Ventures. “For too long, families have faced a system where scientific discoveries move too slowly, clinical trials are difficult to access, and promising therapies often stall before reaching patients. Rare Ventures was created to challenge that reality. By bringing together artificial intelligence, venture philanthropy, therapeutic development, and world-class institutions, we are building a new model designed to accelerate therapies and create hope for millions of families around the world.”
The Richard King Mellon Foundation’s investment will support the establishment of Rare Ventures’ foundational infrastructure, including patient data platforms, basic and translational research programs, therapeutic development capabilities, clinical trial innovation, and strategic partnerships across academia, industry, and healthcare.
“Rare diseases represent one of the greatest unmet challenges in medicine, affecting millions of families while receiving only a fraction of the attention and resources devoted to more common conditions,” said Sam Reiman, Director and Trustee of the Richard King Mellon Foundation. “Rare Ventures brings together an exceptional group of partners around a bold vision: creating the infrastructure, partnerships, and innovation ecosystem needed to accelerate therapies for patients. We believe Pittsburgh possesses the talent, institutions, and collaborative spirit necessary to lead this effort, and we are proud to support its launch.”
Pittsburgh was selected as the home of Rare Ventures because of its unique concentration of strengths across artificial intelligence, life sciences, clinical research, advanced manufacturing, and translational medicine. The platform will collaborate with institutions including the University of Pittsburgh Health Sciences, UPMC Children’s Hospital of Pittsburgh, the UPMC Vision Institute, the Department of Human Genetics, the University of Pittsburgh School of Public Health, Carnegie Mellon University, Stanford Medicine, ElevateBio and industry partners spanning biotechnology and advanced therapeutics.
At the center of the platform is a shared vision: creating a coordinated system capable of transforming scientific breakthroughs into approved therapies at a pace and scale not previously possible.
“Throughout our work with the EB community, we’ve witnessed what becomes possible when patients, researchers, physicians, philanthropists, and industry leaders unite around a common purpose,” said Jill and Eddie Vedder, Co-Founders of the EB Research Partnership, Rock and Roll Hall of Fame inductee, and global advocates for rare disease patients. “Rare Ventures represents the next chapter of that journey. It’s about taking lessons learned from one disease and building something that can help families facing thousands of rare diseases. Every patient deserves hope, and every scientific breakthrough deserves the opportunity to reach the people who need it most. When we think of Pittsburgh, we think of the City of Champions. Thanks to the Richard King Mellon Foundation, the team we have united are truly the champions of the rare disease community”
Rare Ventures will also build upon Curator ™, an AI-powered, genomics-driven patient platform developed through collaborations with leading technology and academic partners. The platform is designed to connect patients with specialists, clinical trials, and research opportunities while providing researchers and industry partners with the data needed to accelerate therapeutic development.
“Rare diseases have long suffered from fragmented data, limited patient populations, and barriers that make research and clinical development extraordinarily difficult,” said Jean Tang, MD, PhD, Professor of Dermatology at Stanford Medicine. “The ability to responsibly connect patient data, genomic insights, clinical expertise, and emerging technologies creates an unprecedented opportunity to accelerate discovery and improve outcomes for patients worldwide.”
The platform will leverage Pittsburgh’s scientific and clinical ecosystem to support therapeutic development across multiple disease areas, including genetic, pediatric, neurological, metabolic, and ophthalmologic conditions.
“Pittsburgh is uniquely positioned to lead the next generation of rare disease innovation,” said Terence Dermody, MD, chair of pediatrics at the University of Pittsburgh School of Medicine and physician-in-chief and scientific director at UPMC Children’s Hospital of Pittsburgh. “By combining world-class clinical expertise, cutting-edge research, and collaborative infrastructure, Rare Ventures has the potential to accelerate the translation of scientific discoveries into therapies that improve and save lives.” The Children’s Hospital team combines the synergistic efforts of Pitt’s basic and translational scientists in the Genetics, Genomics, and Growth Center, led by Associate Professor Arjumand Ghazi and Professor Eric Goetzman, and clinical investigators in the Center for Rare Disease Therapy, led by Jerry Vockley, Professor and Cleveland Family Endowed Chair in Pediatric Research.
The platform’s ophthalmology efforts will build on pioneering work in inherited retinal diseases and vision restoration conducted at Pitt and partner institutions.
“Advances in genetics, regenerative medicine, and precision therapeutics have created extraordinary opportunities for patients affected by inherited retinal diseases and other rare ophthalmic conditions,” said José-Alain Sahel, MD, the Eye & Ear Foundation Endowed Chair of the Department of Ophthalmology at the University of Pittsburgh School of Medicine and director of the UPMC Vision Institute. “Rare Ventures provides a framework for bringing together the scientific, clinical, and translational capabilities needed to move these innovations toward patients more rapidly.”
Artificial intelligence and data science will play a central role in Rare Ventures’ strategy, leveraging Pittsburgh’s position as a global leader in AI research and technology development.
“Carnegie Mellon’s world-class expertise in artificial intelligence, computational biology, genomics, and nucleic acid therapeutics can accelerate the discovery, design, and validation of treatments for rare diseases,” said Barbara Shinn-Cunningham, PhD, Glen de Vries Dean of the Mellon College of Science at Carnegie Mellon University. “CMU researchers are developing AI-driven platforms to identify therapeutic targets, build foundation models for gene therapy design, analyze large-scale genomic data and uncover disease-modifying genetic factors. These therapeutic technologies, along with others under development at CMU, lay the foundation for new approaches to treating a broad range of diseases.”
Rare Ventures will also benefit from Pittsburgh’s growing leadership in advanced biomanufacturing. The Richard King Mellon Foundation has also supported the creation of Pitt BioForge, a $100 million cell and gene therapy biomanufacturing center developed by ElevateBio and the University of Pittsburgh. Together, these complementary investments strengthen Pittsburgh’s position as a national destination for therapeutic innovation, translation, and commercialization.
“The future of medicine depends on our ability to translate scientific breakthroughs into safe and effective treatments that can be available for all” said Christopher Murphy, Chief Executive Officer at ElevateBio. “ElevateBio is proud to support the Rare Ventures initiative and to further Pittsburgh’s growing leadership in advanced therapies and manufacturing, providing expertise, infrastructure, and capacity to realize breakthrough medicines for people and families in need.”
Over time, Rare Ventures aims to create a self-sustaining model in which philanthropic capital, venture philanthropy returns, industry partnerships, and platform-based infrastructure continuously generate resources that can be reinvested into future therapeutic development. The long-term vision is to create a scalable engine capable of accelerating therapies across hundreds of rare diseases and serving as a blueprint for the future of medical innovation.
“Ultimately, our success will not be measured by the model we build, the capital we deploy, or the partnerships we unite,” said Hund. “It will be measured by the therapies we help bring to patients and the lives we improve. That is the mission that unites everyone behind Rare Ventures. We thank the Richard King Mellon Foundation for making it possible.”
About Rare Ventures
Rare Ventures is a first-of-its-kind venture philanthropy platform dedicated to accelerating therapies and cures for rare diseases. Built on the belief that every patient deserves solutions and every breakthrough deserves a path to patients, Rare Ventures brings together artificial intelligence, patient data, basic and translational research, clinical development, regulatory strategy, manufacturing, and commercialization into a single integrated engine for innovation.
Founded by leaders in patient advocacy, business, science, medicine, philanthropy, and biotechnology, Rare Ventures is creating a new model for transforming scientific discoveries into approved treatments: one designed to move faster, scale further, and reinvest success back into future cures. By combining the discipline of venture capital with the mission of philanthropy, Rare Ventures aims to do for rare disease medicine what venture capital did for technology: create the infrastructure, partnerships, and sustainable funding model needed to accelerate innovation at scale.
Headquartered in Pittsburgh and built for the world, Rare Ventures seeks to create a future where no rare disease is too rare to matter.
About the Richard King Mellon Foundation
Founded in 1947, the Richard King Mellon Foundation is the largest foundation in Southwestern Pennsylvania, and one of the 50 largest in the world. The Foundation’s 2025 year-end net assets were $3.3 billion, and its Trustees in 2025 disbursed more than $167 million in grants and program-related investments. The Foundation focuses its funding on six primary program areas, delineated in its 2021-2030 Strategic Plan.
About EB Research Partnership
EB Research Partnership (EBRP) is the world’s largest nonprofit funder dedicated to epidermolysis bullosa research. Through its pioneering venture philanthropy model, EBRP funds high-impact research, invests in therapeutic development, and reinvests financial returns into future cures. EBRP’s mission is to cure EB and create a blueprint for accelerating treatments across rare diseases.
About the University of Pittsburgh Health Sciences
The University looks to its schools of the health sciences to propel scientific discovery and clinical innovation and to educate and prepare future researchers, physicians, health care providers and leaders for 21st-century careers. Pitt Health Sciences is one of the most comprehensive clusters of health sciences schools in the country, boasting schools of medicine, dental medicine, health and rehabilitation sciences, nursing, pharmacy, and public health. The health sciences are a critical part of the epicenter of one of the largest and most prestigious academic and medical research hubs in the nation, comprising six health sciences schools and UPMC–a $30 billion health care provider and insurer. Given the Pitt Health Sciences schools’ close physical proximity to each other and their commitment to collaboration, there is incredible potential for interdisciplinary education and research, as well as integration across the spectrum of research disciplines, from basic to applied research.
All six Pitt Health Sciences schools are highly ranked nationally. In fiscal year 2025, the University of Pittsburgh ranked seventh in the nation for NIH funding with more than 94% of that funding awarded to the health sciences. The University boasts a total annual research budget of more than $1.5 billion.
With nearly 7,000 students, led by 3,600+ expert faculty members, the health sciences are known for collaboration within and across schools. Anchored by the School of Medicine at the University of Pittsburgh, which is consistently ranked among the nation’s top medical schools by U.S. News & World Report and in the top tier for research, the health sciences schools provide rich and engaging environments to learn, collaborate on research and work for students and faculty. The health sciences schools live their mission: To improve the health and well-being of individuals and populations through cutting-edge research, innovative educational programs and community engagement regionally, nationally and globally.
About UPMC
UPMC is a world-renowned, nonprofit health care provider and insurer committed to delivering exceptional, people-centered care and community services. Headquartered in Pittsburgh and affiliated with the University of Pittsburgh Schools of the Health Sciences, UPMC is shaping the future of health through clinical and technological innovation, research, and education. Dedicated to advancing the well-being of our diverse communities, we provide nearly $2 billion annually in community benefits, more than any other health system in Pennsylvania. Our 100,000 employees — including more than 5,000 physicians — care for patients across more than 40 hospitals and 800 outpatient sites in Pennsylvania, New York, and Maryland, as well as overseas. UPMC Insurance Services covers more than 4 million members, providing affordable, high-quality, value-based care. To learn more, visit UPMC.com
About UPMC Children’s Hospital of Pittsburgh
Regionally, nationally, and globally, UPMC Children’s Hospital of Pittsburgh is a leader in the treatment of childhood conditions and diseases, a pioneer in the development of new and improved therapies, and a top educator of the next generation of pediatricians and pediatric subspecialists. With generous community support, UPMC Children’s Hospital has fulfilled this mission since its founding in 1890. UPMC Children’s is recognized consistently for its clinical, research, educational, and advocacy-related accomplishments, including being nationally ranked in all 11 pediatric specialties by U.S. News & World Report.
About The Retinal Dystrophy Clinic at the UPMC Vision Institute
The UPMC Vision Institute’s Retinal Dystrophy Clinic is a national leader in gene-based treatments; the clinic is an approved center for Luxturna, the FDA-approved gene therapy for RPE65-related retinal dystrophy, and participates in numerous clinical trials under the leadership of internationally acclaimed clinician-scientist José-Alain Sahel. The Institute’s Pitt researchers and their collaborators are also at the forefront of emerging therapies, including optogenetics, which has shown early success in restoring functional vision in patients with advanced photoreceptor degeneration, and retinal prosthetics such as the PRIMA chip. Care is delivered by a world-class team of retina specialists, surgeons, geneticists, and rehabilitation experts.
About the Vision Institute
The UPMC Vision Institute harnesses the combined clinical and research expertise of UPMC and the University of Pittsburgh Department of Ophthalmology to prevent vision loss and restore sight. Led by Distinguished Professor and Chair José-Alain Sahel, MD, the Institute’s headquarters at UPMC Mercy Pavilion in Pittsburgh’s Uptown neighborhood spans 410,000 square feet of clinical and research space and houses a dedicated clinical trials center, cutting-edge biotherapy treatments, complete low-vision rehabilitation technologies, and patient-centered resources including a life skills apartment, street lab, and rooftop sensory garden. Its physicians, scientists, and engineers work in close collaboration to move research rapidly into practice, transforming the diagnosis and treatment of conditions such as macular degeneration, glaucoma, diabetic retinopathy, and genetic retinal dystrophies.
About The Eye & Ear Foundation
The Eye & Ear Foundation is a nonprofit organization that supports research and expands access to care by raising philanthropic dollars for the University of Pittsburgh’s Departments of Ophthalmology and Otolaryngology. The Foundation advances discovery and fosters collaboration in vision restoration and rare ophthalmic diseases by accelerating the development of new treatments and cures to improve patient outcomes.
About Carnegie Mellon University
Carnegie Mellon University is a private, internationally ranked research university known for groundbreaking work in the arts, artificial intelligence, robotics, advanced manufacturing and behavioral economics. Its seven schools and colleges support an entrepreneurial spirit and diverse community of scholars, researchers, creators and innovators in Pittsburgh and around the world. CMU takes pride in challenging the curious and passionate to imagine and deliver work that matters.
About ElevateBio
ElevateBio is a technology-driven advanced therapy contract development and manufacturing organization (CDMO) powering the creation of life-transforming therapies. The company helps biopharmaceutical partners design, develop, and manufacture therapies from early discovery through commercialization, combining proprietary gene editing technologies and discovery services, cGMP manufacturing capabilities, and industry-leading expertise to accelerate development across a breadth of therapeutic approaches and modalities. Through continuous investment in automation, AI, and next-generation technologies, ElevateBio delivers the quality, speed, and scale partners need to bring advanced therapies to more patients.